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Orphan Drug Status Granted to Treatment for Relapsed DLBCL

Karyopharm Therapeutics Inc. has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for their lead drug candidate for the treatment of Diffuse Large B-Cell Lymphoma (DLBCL).

Karyopharm Therapeutics is a self-described "clinical-stage pharmaceutical company focused on the discovery and development of novel first-in-class drugs directed against nuclear transport targets for the treatment of cancer and other major diseases."

The drug in question is Selinexor (KPT-330). This is a first-in-class, oral Selective Inhibitor of Nuclear Export (SINE) compound. Selinexor works by binding to the nuclear export protein XPO1 (also called CRM1), leading to the accumulation of tumor suppressor proteins in the cell nucleus. This re-initiates and even amplifies their tumor suppressor function, which in turn is believed to lead to the selective induction of apoptosis (programmed cell death) in cancer cells.

So far, over 300 patients have been treated with Selinexor in Phase 1 and Phase 2 clinical trials in advanced hematologic malignancies and solid tumors.

"The granting of Orphan Drug Designation by the FDA for DLBCL is another significant milestone in the Selinexor development program," said Dr. Sharon Shacham, Founder, CSO and President of Karyopharm. "There are limited treatment options for patients with relapsed or refractory DLBCL, with no new agents approved for this indication over the past two decades. Many patients relapse after responding to multi-agent first-line therapy. The fundamental treatment of DLBCL has changed little in the past two decades, with no new or targeted agents approved for this indication.

"Accordingly, we are excited about the prospects for Selinexor's novel mechanism of action to potentially treat this patient population, either alone or in combination with other therapies."